What Are Desmoid Tumors

  James Cassidy, MD, PhD, Chief Medical Officer at SpringWorks Therapeutics, gives an overview of Desmoid tumors. As Dr. Cassidy explains, Desmoid tumors are tumors that can grow anywhere on the body into surrounding healthy tissues, including joints, muscle, and...

FDA Approves Ruxolitinib to Treat Chronic GVHD

The Food and Drug Administration (FDA) has approved ruxolitinib (Jakafi)  to treat chronic graft-versus-host disease (GVHD) after failure of one or two lines of systemic therapy in patients 12 years and older. Chronic GVHD is a rare complication that can occur after a...

When to Suspect AADC Deficiency in a Baby

  Kylie O’Keefe, Senior Vice President, Head of Global Commercial and Corporate Strategy at PTC Therapeutics, describes early signs and symptoms of aromatic L-amino acid decarboxylase (AADC) deficiency. AADC deficiency is a rare, genetic disorder due to a defect...